Roche Doubles Down on Alzheimer’s, Huntington’s with $60M Ionis Partnership

Pictured: Roche buildings in Basel, Switzerland/iStock, olli0815

Pictured: Roche buildings in Basel, Switzerland/iStock, olli0815

Roche is paying $60 million upfront to longtime partner Ionis Pharmaceuticals for two novel RNA-targeting programs for treating Alzheimer’s and Huntington’s diseases, the companies announced Wednesday.

Under the terms of the partnership, Ionis will advance the therapies through the preclinical stages while Roche will have responsibility for clinical development, manufacturing and commercialization of the medicines if they receive regulatory approval. Ionis receives its upfront payment and “is eligible to receive development, regulatory and commercial milestone payments, and tiered royalties,” the companies said in the press release.

The partnership is intended to “leverage Ionis' expertise discovering medicines that target the root cause of central nervous system diseases” in combination with “Roche's global experience developing and commercializing therapies for nervous system disorders.”

The companies were mum on details of the programs, describing them in the release as “two undisclosed early-stage programs” for RNA-targeting investigational medicines for treating Alzheimer’s and Huntington’s.

A genetic neurodegenerative disease, Huntington’s occurs when the cytosine-adenine-guanine trinucleotide sequence in the HTT gene expands, producing a toxic protein that destroys neurons. While Alzheimer’s is associated with the appearance of protein plaques in the brain that lead to the death of brain cells, no single factor has been identified as the cause of the disease.

The announcement of Wednesday’s deal is just the latest move in a decade-long partnership between the two companies. They first partnered in 2013 on tominersin, an investigational medicine for treatment of Huntington’s disease. Roche is currently conducting a Phase II study on the compound. The companies have expanded that partnership to develop a pre-clinical stage mutant HTT selective antisense oligonucleotide for Huntington’s disease.

In 2018, Roche and Ionis also began work on RG6299, an investigational ligand-ligand antisense medicine for treatment of IgA nephropathy (IgAN)—which is when immunoglobulin A builds up in the kidneys—as well as geographic atrophy (GA), a late-stage dry macular degeneration disease that affects the eyes. Ionis is currently leading a Phase II study of the compound for the treatment of GA, and Roche is leading a Phase III study for the treatment of IgAN.

Roche has been on a bit of a losing streak when it comes to Alzheimer’s disease, however. Last year, Roche’s primary candidate gantenerumab failed to hit its primary endpoints in two separate Phase III studies. Earlier in 2022, the company’s crenezumab—also a monoclonal antibody—failed to slow or prevent a rare form of autosomal-dominant Alzheimer's disease in a Phase III trial.

Connor Lynch is a freelance writer based in Ottawa, Canada. Reach him at lynchjourno@gmail.com.

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